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Blood, 1 October 2008, Vol. 112, No. 7, pp. 2979-2989. Prepublished online as a Blood First Edition Paper on June 27, 2008; DOI 10.1182/blood-2008-03-140830. This Article Full Text Full Text (PDF) All Versions of this Article: blood-2008-03-140830v1 112/7/2979    most recent Alert me when this article is cited Alert me if a correction is posted Citation Map Services Email this article to a friend Similar articles in this journal Similar articles in PubMed Alert me to new issues of the journal Download to citation manager Rights and Permissions Citing Articles Citing Articles via HighWire Citing Articles via CrossRef Citing Articles via Google Scholar Google Scholar Articles by Prasad, V. K. Articles by Kurtzberg, J. Search for Related Content PubMed PubMed Citation Articles by Prasad, V. K. Articles by Kurtzberg, J. Related Collections Transplantation Clinical Trials and Observations Social Bookmarking                   What's this?  Previous Article  |  Table of Contents  |  Next Article  TRANSPLANTATION Unrelated donor umbilical cord blood transplantation for inherited metabolic disorders in 159 pediatric patients from a single center: influence of cellular composition of the graft on transplantation outcomes Vinod K. Prasad1, Adam Mendizabal2, Suhag H. Parikh1, Paul Szabolcs1, Timothy A. Driscoll1, Kristin Page1, Sonali Lakshminarayanan1, June Allison1, Susan Wood1, Deborah Semmel1, Maria L. Escolar3, Paul L. Martin1, Shelly Carter2, and Joanne Kurtzberg1 1 Pediatric Blood and Marrow Transplant Program, Duke University Medical Center, Durham, NC; 2 The EMMES Corporation, Rockville, MD; and 3 Program for Neurodevelopmental Function in Rare Disorders, Center for the Study of Development and Learning at the University of North Carolina Chapel Hill Outcomes of 159 young patients with inherited metabolic disorders (IMDs) undergoing transplantation with partially HLA-mismatched unrelated donor umbilical cord blood were studied to investigate the impact of graft and patient characteristics on engraftment, overall survival (OS), and graft-versus-host disease (GVHD). Patients received myeloablative chemotherapy (busulfan, cyclophosphamide, ATG) and cyclosporine-based GVHD prophylaxis. Infused cell doses were high (7.57 x 107/kg) because of the patients' young age (median, 1.5 years) and small size (median, 12 kg). Median follow-up was 4.2 years (range, 1-11 years). The cumulative incidences of neutrophil and platelet engraftment were 87.1% (95% confidence interval [CI], 81.8%-92.4%) and 71.0% (95% CI, 63.7%-78.3%). A total of 97% achieved high (> 90%) donor chimerism. Serum enzyme normalized in 97% of patients with diseases for which testings exist. Grade III/IV acute GVHD occurred in 10.3% (95% CI, 5.4%-15.2%) of patients. Extensive chronic GVHD occurred in 10.8% (95% CI, 5.7%-15.9%) of patients by 1 year. OS at 1 and 5 years was 71.8% (95% CI, 64.7%-78.9%) and 58.2% (95% CI, 49.7%-66.6%) in all patients and 84.5% (95% CI, 77.0%-92.0%) and 75.7% (95% CI, 66.1%-85.3%) in patients with high (80-100) performance score. In multivariate analysis, favorable factors for OS were high pretransplantation performance status, matched donor/recipient ethnicity, and higher infused colony forming units. CiteULike    Connotea    Del.icio.us    Digg    Reddit    Technorati    What's this? This article has been cited by other articles: J. Tolar, M. Eapen, P. J. Orchard, and B. R. Blazar Acid sphingomyelinase deficiency does not protect from graft-versus-host disease in transplant recipients with Niemann-Pick disease Blood, January 14, 2010; 115(2): 434 - 435. [Full Text] [PDF] L. Ye, J. C. Chang, C. Lin, X. Sun, J. Yu, and Y. W. Kan Induced pluripotent stem cells offer new approach to therapy in thalassemia and sickle cell anemia and option in prenatal diagnosis in genetic diseases PNAS, June 16, 2009; 106(24): 9826 - 9830. [Abstract] [Full Text] [PDF]

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